A phase II, double-blind, exploratory, parallel-group, placebo-controlled clinical study to assess two dosing regimens of GSK2402968 for efficacy, safety, tolerability and pharmacokinetics in ambulant subjects with Duchenne muscular dystrophy

Trial information

This was a Phase IIa, multicentre, double blind, exploratory, parallel clinical trial of GSK2402968 in ambulant DMD boys.

Subcutaneous injections of the drug were given as a continuous OR intermittent regimen over 24 and 48 weeks.

Primary objective

To assess the efficacy of 2 different dosing regimens of GSK2402968,

Secondary objectives

• To assess the safety and tolerability of 2 different doses of the drug
• To assess the pharmacokinetics of the 2 different drug doses over 48 weeks
• To assess long term efficacy of the 2 different drug doses

Recruitment

• 54 subjects
• 2 parallel groups
• Each group included 16 patients on the drug and 8 on matched placebo (2:1 ratio)

This trial recruited in UK at the Great Ormond Street Hospital (GOSH), London and at the Royal Victoria Infirmary, Newcastle.